Pemigatinib

Pemigatinib is a small molecule kinase inhibitor that targets FGFR1, 2 and 3 with IC 50 values of less than 2 nM. Pemigatinib also inhibited FGFR4 in vitro at a concentration approximately 100 times higher than those that inhibit FGFR1, 2, and 3.

• PEMAZYRE is a kinase inhibitor indicated: for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test. ( 1 , 2.1 ) This indication is approved under accelerated approval based on overall response rate and duration of response.

• None. None.contraindicated

Cholangiocarcinoma: Confirm the presence of an FGFR2 fusion or rearrangement prior to initiation of treatment with PEMAZYRE. ( 2.1 ) Recommended dosage is 13.5 mg orally once daily for 14 consecutive days followed by 7 days off therapy in 21-day cycles. Continue treatment until disease progression or unacceptable toxicity occurs. ( 2.2 ) Myeloid/lymphoid neoplasms with FGFR1 rearrangement: Recommended dosage is 13.5 mg orally once daily. Continue treatment until disease progression or unacceptable toxicity. ( 2.2 ) All patients treated with PEMAZYRE: Swallow tablet whole, with or without food. ( 2.2 ) Severe Renal Impairment: the recommended dosage of PEMAZYRE is 9 mg with the schedule (intermittent or continuous) designated for the indication. ( 2.5 , 8.6 , 12.3 ) Severe Hepatic Impairment: the recommended dosage of PEMAZYRE is 9 mg with the schedule (intermittent or continuous) designated for the indication. ( 2.6 , 8.7 , 12.3 ) 2.1 Patient Selection Select patients for the treatment of locally advanced or metastatic cholangiocarcinoma with PEMAZYRE based on the presence of an FGFR2 fusion or rearrangement as detected by an FDA-approved test [see Clinical Studies ( 14.1 )]. Information on FDA-approved test(s) for the detection of an FGFR2 fusion or rearrangement in cholangiocarcinoma is available at http://www.fda.gov/CompanionDiagnostics . Select patients for the treatment of relapsed or refractory myeloid/lymphoid neoplasms with FGFR1 rearrangement with PEMAZYRE based on the presence of an FGFR1 rearrangement [see Clinical Studies ( 14.2 )] .

Ocular Toxicity : PEMAZYRE can cause retinal pigment epithelial detachment. Perform ophthalmological examination including optical coherence tomography (OCT) prior to initiation of therapy, every 2 months for the first 6 months of treatment and every 3 months thereafter, and urgently at any time for visual symptoms. ( 2.3 , 5.1 ) Hyperphosphatemia and Soft Tissue Mineralization : PEMAZYRE can cause hyperphosphatemia leading to soft tissue mineralization, cutaneous calcification, calcinosis, and non-uremic calciphylaxis. Monitor for hyperphosphatemia and withhold, reduce the dose, or permanently discontinue based on duration and severity of hyperphosphatemia. ( 2.3 , 5.2 ) Embryo-Fetal Toxicity : Can cause fetal harm. Advise patients of reproductive potential of the potential risk to the fetus and use effective contraception. ( 5.3 , 8.1 , 8.3 ) 5.1 Ocular Toxicity Retinal Pigment Epithelial Detachment (RPED) PEMAZYRE can cause RPED, which may cause symptoms such as blurred vision, visual floaters, or photopsia. Clinical trials of PEMAZYRE did not conduct routine monitoring including optical coherence tomography (OCT) to detect asymptomatic RPED; therefore, the incidence of asymptomatic RPED with PEMAZYRE is unknown. Among 635 patients who received a starting dose of PEMAZYRE 13.5 mg across clinical trials, RPED occurred in 11% of patients, including Grade 3-4 RPED in 1.3%. The median time to first onset of RPED was 56 days.

The following adverse reactions are discussed elsewhere in the labeling: Ocular Toxicity [see Warnings and Precautions ( 5.1 )] Hyperphosphatemia and Soft Tissue Mineralization [see Warnings and Precautions ( 5.2 )] Cholangiocarcinoma: The most common adverse reactions (incidence ≥ 20%) are hyperphosphatemia, alopecia, diarrhea, nail toxicity, fatigue, dysgeusia, nausea, constipation, stomatitis, dry eye, dry mouth, decreased appetite, vomiting, arthralgia, abdominal pain, hypophosphatemia, back pain, and dry skin. ( 6.1 ) Myeloid/lymphoid neoplasms with FGFR1 rearrangement: The most common (≥ 20%) adverse reactions are hyperphosphatemia, nail toxicity, alopecia, stomatitis, diarrhea, dry eye, fatigue, rash, abdominal pain, anemia, constipation, dry mouth, epistaxis, serous retinal detachment, extremity pain, decreased appetite, dry skin, dyspepsia, back pain, nausea, blurred vision, peripheral edema, and dizziness. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Incyte Corporation at 1-855-463-3463 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

Lactation: Advise not to breastfeed. ( 8.2 ) 8.1 Pregnancy Risk Summary Based on findings in an animal study and its mechanism of action, PEMAZYRE can cause fetal harm or loss of pregnancy when administered to a pregnant woman [see Clinical Pharmacology ( 12.1 )] . There are no available data on the use of PEMAZYRE in pregnant women. Oral administration of pemigatinib to pregnant rats during the period of organogenesis at maternal plasma exposures below the human exposure at the clinical dose of 13.5 mg resulted in fetal malformations, fetal growth retardation, and embryo-fetal death (see Data) . Advise pregnant women of the potential risk to a fetus. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Data Animal Data Once daily oral administration of pemigatinib to pregnant rats during the period of organogenesis resulted in 100% embryofetal mortality due to post-implantation loss at doses ≥ 0.3 mg/kg (approximately 0.6 times the human exposure based on AUC at the clinical dose of 13.5 mg).

Metabolism

The geometric mean (CV%) steady-state pemigatinib AUC 0-24h was 2620 nM·h (54%) and C max was 236 nM (56%) for 13.5 mg orally once daily. Steady state pemigatinib concentrations increased proportionally over the dose range of 1 to 20 mg (0.07 to 1.5 times the recommended dose).

• Apr 17, 2020NDANDA213736Incyte Corp

1. 1Off Label Use14018%
2. 2Disease Progression13617%
3. 3Death8110%
4. 4Fatigue769.6%
5. 5Alopecia607.6%
6. 6Cholangiocarcinoma597.5%
7. 7Hyperphosphataemia516.5%
8. 8Drug Ineffective465.8%
9. 9Diarrhoea425.3%
10. 10Nausea394.9%
11. 11Constipation384.8%
12. 12Hospitalisation374.7%
13. 13Decreased Appetite354.4%
14. 14Stomatitis324.1%
15. 15Dry Mouth313.9%

The 10 most recently updated of 48 ClinicalTrials.gov registrations naming Pemigatinib as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Safety and Efficacy of Retifanlimab (INCMGA00012) Alone or in Combination With Other Therapies in Participants With Advanced or Metastatic Endometrial Cancer Who Have Progressed on or After Platinum-based Chemotherapy.
  Completed · Phase 2 · Interventional · 206 enrolled · Incyte Corporation
  NCT04463771updated 2026-06-11
• PH 2 Pemigatinib in SDH-deficient GIST
  Recruiting · Phase 2 · Interventional · 24 enrolled · Dana-Farber Cancer Institute
  NCT07434843updated 2026-05-22
• Pemigatinib + Afatinib in Advanced Refractory Solid Tumors
  Recruiting · Phase 1 · Interventional · 70 enrolled · Massachusetts General Hospital
  NCT06302621updated 2026-05-15
• A Phase II Study of Pemigatinib Plus Durvalumab in Previously Treated Advanced Intrahepatic Cholangiocarcinoma Patients With FGFR-2 Fusion or Rearrangement
  Recruiting · Phase 2 · Interventional · 38 enrolled · Mehmet Akce
  NCT06728410updated 2026-05-08
• A Phase II Nationwide, Fully Decentralized, Telemedicine Study of Pemigatinib in Adult Patients With Advanced or Metastatic Pancreatic Cancer With FGFR Genetic Alterations
  Recruiting · Phase 2 · Interventional · 40 enrolled · Sameek Roychowdhury
  NCT06906562updated 2026-02-17
• Phase I/II Trial of Pemigatinib in Combination With Atezolizumab and Bevacizumab for Treatment of Advanced Cholangiocarcinoma With FGFR2 Fusion
  Recruiting · Phase 1 · Phase 2 · Interventional · 25 enrolled · M.D. Anderson Cancer Center
  NCT06439485updated 2026-01-14
• Study to Evaluate the Efficacy and Safety of Pemigatinib in Participants With Previously Treated Glioblastoma or Other Primary Central Nervous System Tumors Harboring Activating FGFR1-3 Alterations
  Terminated · Phase 2 · Interventional · 83 enrolled · Incyte Corporation
  NCT05267106updated 2025-12-30
• Pemigatinib After Chemotherapy for the Treatment of Newly Diagnosed Acute Myeloid Leukemia
  Recruiting · Phase 1 · Interventional · 32 enrolled · OHSU Knight Cancer Institute
  NCT04659616updated 2025-12-19
• Pemigatinib in Patients With Relapsed or Refractory B-cell Non-Hodgkin Lymphomas
  Recruiting · Phase 2 · Interventional · 27 enrolled · University of Utah
  NCT06300528updated 2025-12-03
• A Phase II Randomized Study Comparing the Efficacy and Safety of Targeted Therapy or Cancer Immunotherapy Versus Platinum-Based Chemotherapy in Patients With Cancer of Unknown Primary Site
  Completed · Phase 2 · Interventional · 529 enrolled · Hoffmann-La Roche
  NCT03498521updated 2025-11-19

Frequently asked questions

How does Pemigatinib work?

Pemigatinib is a small molecule kinase inhibitor that targets FGFR1, 2 and 3 with IC 50 values of less than 2 nM. Pemigatinib also inhibited FGFR4 in vitro at a concentration approximately 100 times higher than those that inhibit FGFR1, 2, and 3.

What is Pemigatinib used for?

According to FDA labeling, Pemigatinib carries indications including: PEMAZYRE is a kinase inhibitor indicated: for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test. ( 1 , 2.1 ) This indication is approved under accelerated approval based on overall response rate and duration of response.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Pemigatinib?

Pemigatinib is classified as Fibroblast growth factor receptor (FGFR) tyrosine kinase inhibitors, Kinase Inhibitor, Kinase Inhibitors, Multidrug and Toxin Extrusion Transporter 1 Inhibitors, Organic Cation Transporter 2 Inhibitors, P-Glycoprotein Inhibitors, Cellular Proliferation Alteration.

What are the brand names for Pemigatinib?

Pemigatinib is marketed under brand names including Pemazyre.

What are the contraindications for Pemigatinib?

Pemigatinib labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.